Gene fix may cure infants with no immune system
NCT ID NCT01512888
First seen Nov 10, 2025 · Last updated May 24, 2026 · Updated 30 times
Summary
This study tests a gene therapy for infants with X-linked severe combined immunodeficiency (SCID-X1), a life-threatening condition where babies have no working immune system. Doctors take the baby's own bone marrow stem cells, add a healthy copy of the faulty gene, and return them to the body after mild chemotherapy. The goal is to give these infants a normal, lasting immune system without needing a stem cell donor.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Seattle Children's Research Institute
Seattle, Washington, 98101, United States
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St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
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University of California-San Francisco
San Francisco, California, 94158, United States
Conditions
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